Multiple Federal Agencies to Attend Discussion with Community Stakeholders on Living with Duchenne Muscular Dystrophy
(March 05, 2018)

HACKENSACK, N.J., March 5, 2018 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), will host the Duchenne Patient-Focused Compass Meeting: Navigating our Pathway Forward as part of the organization's Annual Advocacy Conference. The meeting will be held on Monday, March 5, 2018 at the Mayflower Hotel in Washington, DC from 10 AM – 4 PM. The Duchenne Patient-Focused Compass meeting will be comprised of panels and presentations with Duchenne community members (patients and caregivers) to explore 'living with Duchenne;' clinical trial and therapeutic experiences; and access to services, supports, and approved therapies across the Duchenne spectrum.

Following a discussion with key FDA personnel last fall, PPMD worked on developing a meeting format that utilizes the "FDA PFDD Workshop" framework, while also broadening the day's scope to apply to federal partners and stakeholders beyond FDA. This meeting will allow the Duchenne community to build upon the Duchenne Guidance, Patient Preferences studies, and formalized data collection that were previously shared with the FDA. Invited members of FDA, NIH, CDC, CMS, and other federal agencies, along with relevant policy stakeholders will be present. Live polling of Duchenne families present and those viewing the meeting online will provide real time data about patient and caregiver perspectives and experiences.

In preparing for this meeting, PPMD invited all of the Duchenne community foundation partners to join the Community Advisory Board (CAB) for the meeting. Currently 14 Duchenne foundations have signed onto the CAB.

CAB members include:

Best Day Ever Foundation

Coalition Duchenne

Foundation for a Future

Foundation to Eradicate Duchenne (FED)

Hope for Gus

Hope for Javier

JB's Keys

Jett Foundation

Little Hercules Foundation

Powers Promise

Ryan's Quest

Team Joseph

Walking Strong

Zach Heger Foundation

PPMD is grateful to all foundations participating in this effort.

In preparation for this important meeting, PPMD published a White Paper intended to reflect the current Duchenne landscape and create context for the March meeting. The White Paper was led by PPMD with the collaboration of the CAB. A meeting report will be published following the March 5 meeting.

Why this Meeting? Why Now?

Since the passage of PDUFA V (during the reauthorization of and the launch of the Patient Focused Drug Development (PFDD) Initiative at the FDA), the agency has been holding a series of disease specific "Voice of the Patient" meetings with different patient communities. These meetings have followed a set "PFDD workshop formula" in order to elicit an understanding of a given disease area and to collect high-level information on burden of disease and symptom management.

In 2017, PPMD's Duchenne Drug Development Roundtable (DDDR) partners worked together to evaluate our research and 'regulatory learnings' throughout 2015 and 2016. PPMD also convened meetings that explored the role of inflammation in Duchenne and the burden of clinical trial participation.

In addition, as a result of our community's efforts around the MD-CARE Act Amendments of 2014 and a strong collaboration between the Centers for Disease Control and Prevention (CDC) and the Duchenne community, the Duchenne/Becker Care Considerations have been updated to reflect changing care standards. The DBMD Care Considerations were published last month (January 2018).

Families unable to attend in person can be part of this historic day by joining the meeting via live streaming. By registering, families can participate in real-time polling/surveying throughout the meeting. To sign up to be a virtual attendee, please register here.

We demand optimal care standards and ensure every family has access to expert healthcare providers, cutting edge treatments, and a community of support. We invest deeply in treatments for this generation of Duchenne patients and in research that will benefit future generations. Our advocacy efforts have secured hundreds of millions of dollars in funding and won two FDA approvals.

Everything we do—and everything we have done since our founding in 1994—helps those with Duchenne live longer, stronger lives. We will not rest until we end Duchenne for every single person affected by the disease. Join our fight against Duchenne at EndDuchenne.org. Follow PPMD on Facebook, Twitter, and YouTube.